Mirdametinib

Merck KGaA to Acquire SpringWorks Therapeutics in $3.9 Billion Deal for Rare Disease Portfolio

25 days ago

• German pharmaceutical giant Merck KGaA has agreed to acquire Connecticut-based SpringWorks Therapeutics for $3.9 billion, paying $47 per share in cash, representing a 26% premium over SpringWorks' unaffected 20-day average price. • The acquisition gives Merck KGaA immediate access to SpringWorks' two FDA-approved rare disease treatments: Ogsiveo for desmoid tumors and Gomekli for neurofibromatosis type 1-related tumors. • The transaction, expected to close in the second half of 2025 pending regulatory approvals, aligns with Merck KGaA's strategic expansion in the U.S. market and is projected to contribute to earnings by 2027.

SpringWorks Anticipates CHMP Opinion on Nirogacestat for Desmoid Tumors in Q2 2025

a month ago

• SpringWorks Therapeutics expects the European Medicines Agency's CHMP to issue an opinion on nirogacestat for adult desmoid tumors in Q2 2025, potentially expanding access beyond its current FDA approval. • Nirogacestat, marketed as OGSIVEO in the US, is an oral gamma secretase inhibitor and the first FDA-approved therapy specifically for adults with desmoid tumors, addressing a significant unmet need. • SpringWorks continues to build its rare disease portfolio, which includes GOMEKLI (mirdametinib) for neurofibromatosis type 1, while advancing additional targeted therapies for solid tumors and hematological cancers.

Biotech Deal Landscape: February-March 2025 Sees Surge in Partnerships Across Multiple Therapeutic Areas

3 months ago

• The first quarter of 2025 witnessed significant biotech partnership activity, with Eli Lilly, AstraZeneca, and Novo Nordisk emerging as top collaborators in deals worth billions across small molecules, antibodies, and RNA therapeutics. • February 2025 featured notable acquisitions including Novartis's $2.15 billion buyout of Anthos Therapeutics, while March saw AstraZeneca acquire Belgian biotech EsoBiotec and Bristol Myers Squibb purchase 2seventy bio for $286 million. • Obesity therapeutics gained significant traction in March 2025, with AbbVie entering the field through a $350 million upfront deal with Gubra for an amylin analog, while Roche partnered with Zealand Pharma on petrelintide in a deal worth up to $5.25 billion.

Olverembatinib Shows Promise in Heavily Pretreated Chronic Myeloid Leukemia

3 months ago

• Phase 1b trial data demonstrates olverembatinib's safety and efficacy in treating patients with heavily pretreated chronic-phase chronic myeloid leukemia, marking a potential new era in CML management. • Dr. Elias Jabbour of MD Anderson Cancer Center highlights olverembatinib as "one of the best TKIs available for CML," emphasizing its strong safety and efficacy profile. • The ongoing registrational phase 3 POLARIS-2 trial (NCT06423911) is now enrolling patients to further evaluate olverembatinib's potential in CML treatment.

SpringWorks' Gomekli Approval Showcases New Model for Rescuing Shelved Rare Disease Drugs

3 months ago

• SpringWorks Therapeutics recently secured FDA approval for Gomekli (mirdametinib), a drug once abandoned by Pfizer, providing the first treatment option for adults with neurofibromatosis type 1. • The Children's Tumor Foundation played a crucial role in rescuing the drug from Pfizer's intellectual property vault, demonstrating how patient advocacy groups can bridge the gap between academia and industry for rare disease treatments. • Experts estimate that approximately 30 more potential treatments for neurofibromatosis alone may be sitting idle in pharmaceutical companies' portfolios, highlighting a significant opportunity to replicate this model across rare diseases.

FDA Grants Priority Review to Troriluzole for Spinocerebellar Ataxia Treatment

3 months ago

• Biohaven's troriluzole has received FDA Priority Review for treating spinocerebellar ataxia (SCA), potentially becoming the first FDA-approved treatment for this rare genetic neurodegenerative disease. • Clinical trials demonstrated troriluzole slowed SCA disease progression by 50-70% over three years, representing a 1.5-2.2 year delay in disease advancement compared to controls. • The FDA's decision on the New Drug Application is expected in Q3 2025, with Biohaven prepared to commercialize troriluzole in the US by 2025 if approved.

Merck KGaA in Advanced Talks to Acquire SpringWorks Therapeutics; BridgeBio Secures EU Approval for ATTR Drug

3 months ago

• Merck KGaA confirms advanced acquisition discussions with SpringWorks Therapeutics, though critical conditions remain unmet and no binding agreement has been reached. • BridgeBio Pharma's Beyonttra (acoramidis) receives European Commission approval for transthyretin amyloidosis with cardiomyopathy, triggering a $75 million milestone payment from Bayer. • Biohaven's troriluzole receives FDA priority review for spinocerebellar ataxia, positioning it to potentially become the first approved treatment for this neurodegenerative disorder.

FDA Approves Brentuximab Vedotin Combination for Relapsed/Refractory Large B-Cell Lymphoma

4 months ago

• The FDA has approved brentuximab vedotin in combination with lenalidomide and rituximab for adult patients with relapsed or refractory LBCL. • The approval is specifically for those ineligible for auto-HSCT or CAR T-cell therapy after two or more lines of systemic therapy. • Data from the ECHELON-3 trial demonstrated a statistically significant improvement in overall survival with the brentuximab vedotin combination. • Common adverse events included neutropenia, thrombocytopenia, anemia, and peripheral neuropathy, manageable with dose modifications.

Daratumumab Plus VRd Improves Outcomes in Transplant-Ineligible Multiple Myeloma Patients

6 months ago

• The phase 3 CEPHEUS trial demonstrated that adding daratumumab to VRd significantly improved outcomes for transplant-ineligible newly diagnosed multiple myeloma patients. • The quadruplet regimen achieved a 60.9% minimal residual disease (MRD) negativity rate compared to 39.4% with VRd alone, demonstrating a significant increase in treatment depth. • Progression-free survival was also significantly improved with the daratumumab regimen, showing a 43% reduction in the risk of disease progression or death. • These results suggest that daratumumab plus VRd could become a new standard of care for transplant-ineligible multiple myeloma patients, offering improved disease control.

FDA Approves SpringWorks' Gomekli (Mirdametinib) for Neurofibromatosis Type 1

6 months ago

• The FDA has approved Gomekli (mirdametinib) for both adult and pediatric patients with Neurofibromatosis Type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection. • Approval was based on Phase 2b ReNeu trial data, demonstrating a 41% ORR in adults and 52% in children, alongside deep and durable tumor volume reductions and manageable safety profiles. • Gomekli is the first and only medicine approved for both adults and children with NF1-PN, addressing a significant unmet need, particularly for adult patients who previously lacked approved treatments. • SpringWorks received a rare pediatric disease priority review voucher from the FDA, potentially expediting the review of future drug candidates.

FDA Approves Supernus' ONAPGO for Motor Fluctuations in Advanced Parkinson's Disease

7 months ago

• The FDA has approved ONAPGO (apomorphine hydrochloride) as the first subcutaneous apomorphine infusion device for treating motor fluctuations in advanced Parkinson's disease. • ONAPGO, a wearable device, provides continuous subcutaneous apomorphine infusion, offering more consistent control of OFF time for Parkinson's patients. • Clinical trials demonstrated that ONAPGO significantly reduced daily OFF time by 2.6 hours compared to placebo, with improvements seen as early as week 1. • Supernus plans to launch ONAPGO in the U.S. in the second quarter of 2025, supported by a dedicated nurse education program and access support.

Cell Therapy and Targeted Therapies Dominate Oncology Advances in Early 2025

7 months ago

• The FDA issued a CRL for Atara Biotherapeutics' tabelecleucel due to third-party manufacturing issues, not efficacy or safety data, delaying potential approval for EBV+ PTLD. • EsoBiotec dosed the first patient in a trial for ESO-T01, an in vivo BCMA-directed CAR-T therapy for multiple myeloma, aiming for lower costs and simplified administration. • Obecabtagene autoleucel (obe-cel) gained FDA approval for relapsed/refractory B-cell precursor ALL, offering a less toxic CD19-directed CAR T-cell therapy option. • Arlocabtagene autoleucel (arlo-cel) shows promise in heavily pretreated relapsed/refractory multiple myeloma, eliciting a 48% complete response rate in phase 1 studies.

Drug Development Updates